“Imagine CRISPR cures” is the title of the keynote by Fyodor Urnov at the World CRISPR Day conference on October 20. The talk was far from a celebration: “The fact that editing represents an approach to the majority of monogenic diseases in principle doesn’t mean that some biotech will take on disease number 823 in practice, and there are over 5,000 monogenic conditions on OMIM. Three years to IND in the best case scenario and cost scale of more than $6m per disease, that’s incompatible with either the promise of CRISPR to edit any given mutation which it can do or the unmet medical needs”. Don’t miss the on-demand video to learn more about the challenge of N=1 trials and Urnov’s call to arm against ultra-rare diseases.
Here you can read a selection of notable comments about the landmark paper on in vivo genome-editing published in the New England Journal of Medicine on 26 June. The trial, conducted in the UK and New Zealand, produced the first-ever clinical data supporting the safety and efficacy of intravenous infusion of a single-dose CRISPR treatment. The treatment, developed by two US-based companies (Intellia Therapeutics and Regeneron Pharmaceuticals) targets a rare and fatal condition called transthyretin amyloidosis.
Jennifer Doudna (CRISPR co-inventor and co-founder of Intellia): “It’s a critical first step in being able to inactivate, repair, or replace any gene that causes disease, anywhere in the body” (source Science).Continue reading
According to his Twitter bio, Charles Murray is a “Husband, father, social scientist, writer, Madisonian. Or maybe right-wing ideologue, pseudoscientist, evil. Opinions differ.” You may remember him as the co-author of the controversial book “The Bell Curve” (1994), discussing purported connections between race and intelligence. The bad news is that he recently joined the CRISPR debate by tweeting “Gene editing to raise IQ will have a huge market”. The good news is that confutation is easy and a little irony is the best reply (check out Fyodor Urnov’s tweet in the gallery below).
This is an issue for all tastes and interests. Don’t miss (Broken) Promises of Sustainable Food and Agriculture through New Biotechnologies by Todd Kuiken, Rodolphe Barrangou and Khara Grieger; A Code of Ethics for Gene Drive Research by George Annas and other members of the Controlling and Countering Gene Editing in Mosquitoes research project funded by the DARPA Safe Genes program; The Cas9 Hammer and the Sickle by Fyodor Urnov.
Hopefully, CRISPR-based diagnostics will make an early debut amid COVID-19 outbreak. But what about a CRISPR prophylactic strategy to combat coronaviruses? The proof of concept is here, in bioRxiv, but it will be deployed in the next pandemic if we are lucky. It’s called PAC-MAN, like the videogame, stands for Prophylactic Antiviral CRISPR in huMAN cells, and comes from the Stanley Qi Lab.Continue reading
The year 2019 ended with three years in jail sentenced to He Jiankui for illegal medical practice. The CRISPR-baby scandal’s epilogue was applauded on twitter by a few leading scientists such as Craig Venter and Fyodor Urnov and decried on STAT News by the controversial biohacker Josiah Zayner. Most experts, however, stayed silent.
As stressed by the Washington Post, “the judicial proceedings were not public, and outside experts said it is hard to know what to make of the punishment without the release of the full investigative report or extensive knowledge of Chinese law and the conditions under which He will be incarcerated.”
Great piece of science by the Liu Lab in Nature, describing the brand new “Search-and-replace genome editing without double-strand breaks or donor DNA”. How is the CRISPR community reacting?
Best quote: “One of those ‘Yay, science!!!’ kind of moments” (Fyodor Urnov quoted in Science)
Most ironic: “Congratulations @davidrliu. We’d probably have published this paper as well (The CRISPR Journal tweet)
Best title: Genome Editing Heads to Primetime (Genetic Engineering and Biotechnology News)
Most understated: A New Gene Editing Tool Could Make CRISPR More Precise (Smithsonian Mag)
Most hyped: A New CRISPR Technique Could Fix Almost All Genetic Diseases (Wired)
Doudna meets GSK: University of California CRISPR researchers form drug discovery alliance with pharma giant (source Science)
From transposons to gene therapy: Hijack of CRISPR defences by selfish genes holds clinical promise, according to Fyodor Urnov (Nature News&Views)
Disaster waiting to happen: Russian biologist plans more CRISPR-edited babies (Nature’s editorial and news)